News

So-called silent gene mutations in the cystic fibrosis (CF)-causing CFTR gene are not necessarily silent at all. A research team discovered that one such mutation also causes the resulting protein to have a poor function. The insights put the spotlight on the complexity of CF genetic causes, but also advances…

Abiding by the extensive treatment regimen for cystic fibrosis (CF) can be especially challenging for pediatric patients. A new study found that frequent monitoring of pulmonary function tests (PFTs) and medication reminders can help pediatric CF patients better meet their daily medication and vitamin…

Celtaxsys announced it has completed enrollment of its Phase 2b clinical trial evaluating the company’s lead anti-inflammatory candidate, oral acebilustat, in adult patients with cystic fibrosis (CF). Acebilustat (formerly known as CTX-4430) is a once-daily, oral anti-inflammatory being evaluated for its safety and efficacy in treating diseases like CF. It is a new small…

X-Chem Pharmaceuticals and Vertex Pharmaceuticals will jointly develop small molecules targeting cystic fibrosis (CF) and other severe genetic diseases. Under the collaboration announced May 16, X-Chem will apply its proprietary DEX libraries — which holds more than 120 billion small compounds — to discover new drug candidates against targets involved in CF…

The U.S. Food and Drug Administration (FDA) has expanded its approval of Kalydeco (ivacaftor) to include cystic fibrosis (CF) patients with one of 23 additional rare mutations — more than tripling the number of mutations the oral drug may be used to treat from 10 to 33. But the decision’s…

Spyryx Biosciences will present two studies in Washington this week on SPX-101‘s ability to treat cystic fibrosis by clearing mucus from airways. One of the preclinical-trial studies it will discuss at the American Thoracic Society‘s annual convention showed that SPX-101 improved survival in several models of CF-like lung diseases.

Dalton and Arch Biopartners are partnering to manufacture AB569 for clinical trials involving cystic fibrosis (CF) patients with chronic, antibiotic-resistant bacterial lung infections. Under the agreement, the two will divide manufacturing tasks, including formulating the compound, putting it in containers safely, and doing quality-control testing. The drug will be…

A substance found in a Japanese cypress tree can deliver iron to cells that are unable to obtain it because of faulty proteins, a finding that has implications for cystic fibrosis, according to a study. Researchers said their molecular-prosthetics approach could not only address cystic fibrosis (CF), but also other conditions…

Researchers at TGV-inhalonix said their company’s drug, Mul-1867, has a powerful effect against clinical isolates of fungi in patients with cystic fibrosis (CF) and other severe lung infections. The study “In vitro activity of a novel compound, Mul-1867, against clinically significant fungi Candida spp. and Aspergillus spp.” appeared in the International Journal of Antimicrobial…

Mutated mice that inhaled SPX-101 lived longer, according to a new preclinical study that highlights the drug’s potential to clear mucus and hydrate airways in patients with cystic fibrosis (CF). The study, “SPX-101 is a Novel ENaC-targeted Therapeutic for Cystic Fibrosis that Restores Mucus Transport,” appeared in the American Journal of…